Title : AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.

Pub. Date : 2018 Sep 5

PMID : 30007561






1 Functional Relationships(s)
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1 Huntington"s disease (HD) is a fatal neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the huntingtin gene. trinucleotide huntingtin Homo sapiens